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EU's CHMP Clears Europe's First Gene Therapy, UniQure's Glybera

 
• By John Davis

After three previous negative votes, the Netherlands biotech uniQure gains the first positive opinion from the Committee for Medicinal Products for Human Use for a gene therapy in Europe. Glybera is used to treat the ultra-rare condition lipoprotein lipase deficiency.

In a stunning reversal of fortune for gene therapy, the EU's scientific advisory panel, the Committee for Medicinal Products for Human Use (CHMP), has recommended approval of uniQure NV's Glybera (alipogene tiparvovec), just months after the company's predecessor, Amsterdam Molecular Therapeutics BV (AMT), was taken private following two previous negative CHMP votes. A further CHMP vote, in April 2012, also went against the therapy.

Glybera is the first gene therapy to be recommended for approval in Europe. Such therapies have the potential to cure genetic disorders by replacing a defective gene with a working copy, but have proven extremely difficult to bring to market

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